FDA delays decision on extending Yutrepia to treat PH-ILD
Inhaled form of treprostinil, tentatively OK for PAH, is subject of litigation
Note: This story was updated Jan. 30, 2024, to correct the approved indications of available treprostinil formulations.
The U.S. Food and Drug Administration (FDA) has delayed its decision to extend the use of Yutrepia — Liquidia‘s inhaled dry powder formulation of treprostinil — to people with pulmonary hypertension (PH) associated with interstitial lung disease (PH-ILD).
Yutrepia was given tentative FDA approval in 2021 as a treatment for pulmonary arterial hypertension (PAH). In September 2023, the agency agreed to review the company’s request to extend the therapy’s use to include PH-ILD. The request came in the form of an amended new drug application, or NDA.
The review process needed to confirm an expanded label is ongoing, the FDA recently informed the company.
While the agency did not ask for additional clinical data to support Liquida’s request, it said it could not meet the Jan. 24 date set to decide whether to add PH-ILD to the therapy’s label. It did not issue a new decision date.
United Therapeutics sees Yutrepia as violating patents for Tyvaso
“We are in active communication with the FDA regarding the process we followed to amend our NDA to add PH-ILD to the labeled indication,” Roger Jeffs, PhD, CEO of Liquidia, said in a company press release. “We remain committed to addressing the unmet needs across all patients whose lives may be improved by the unique benefits of YUTREPIA.”
PH is marked by high blood pressure in the blood vessels that pass through the lungs, called the pulmonary arteries. It can be triggered by various conditions, including a narrowing of the pulmonary arteries in PAH, or lung scaring in PH-ILD.
Yutrepia is a formulation of treprostinil — a vasodilator that lowers blood pressure by widening and relaxing the blood vessels — inhaled directly into the lungs via a palm-sized device.
The FDA has approved several formulations of treprostinil, including oral Orenitram and infusion/injection Remodulin for PAH, and inhaled Tyvaso for both PAH and PH-ILD. All three of these formulations are sold by United Therapeutics.
Yutrepia’s tentative approval for PAH meant it has met the safety and efficacy standards needed for approval. However, United believes that Yutrepia violated several patents covering Tyvaso and sued Liquidia. Due to the ongoing litigation, Yutrepia’s final approval cannot yet be granted.
“As communicated by the tentative approval to treat PAH, YUTREPIA has already met the regulatory standards for quality, safety and efficacy,” Jeffs said.
The FDA accepted Liquidia’s request to extend Yutrepia indication to PH-ILD without additional clinical studies. If the agency eventually grants the application, it could come into effect after March 31, when the regulatory exclusivity period protecting Tyvaso from market competition expires.
“Whether the NDA is amended or supplemented, we will continue to prepare for the final FDA approval of Yutrepia to treat both PAH and PH-ILD,” Jeffs noted.
Open-label study underway testing Yutrepia in about 60 PH-ILD patients
The open-label INSPIRE Phase 3 clinical trial (NCT03399604) supported Yutrepia’s tentative approval for PAH. The therapy was generally well tolerated and improved exercise endurance and quality of life.
Liquidia also is enrolling about 60 PAH or PH-ILD adults in its open-label Phase 3 clinical study (NCT04691154) of L606, a sustained-release formulation of treprostinil given with a nebulizer twice daily. Eligible PAH patients have never been treated with prostacyclins (treprostinil-like medications) or are transitioning from Tyvaso or Tyvaso DPI. Patients with PH-ILD also are switching from one of these two medications.